<scp>ABO</scp> blood barrier to engraftment after allogeneic stem cell transplantation in sickle cell disease: A case‐story with two successive <scp>HLA</scp>‐matched sibling donors

Redjoul, Rabah; Beckerich, Florence; Luna, Gonzalo De; Leclerc, Mathieu; Hebert, Nicolas; Sloma, Ivan; Menouche, Dehbia; Bartolucci, Pablo; Maury, Sébastien

doi:10.1002/ajh.26808

Cited by 2 publications

(1 citation statement)

References 14 publications

(36 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“… 208 Moreover, SCD patients requiring allo‐HSCT transplantation often lack suitable donors and are at risk of secondary malignancy after allo‐HSCT transplantation. 209 , 210 , 211 In contrast to allo‐HSCT, autologous HSCs are easy to obtain and have a high rate of transplantation success, but have the potential to lead to disease recurrence. 212 In preclinical studies of SCD, CRISPR–Cas9‐based gene editing has mainly focused on the in vitro genetic modification of autologous hematopoietic stem/progenitor cells (HSPCs) from patients with SCD, designed to perform a one‐time genetic modification on HSPCs of patient origin, which avoids the requirement for allogeneic healthy donor HSPCs and produces corrected blood cells in the later life of patients with SCD without immunological complications.…”

Section: Gene Therapy In Human Diseasesmentioning

confidence: 99%

CRISPR technology in human diseases

Feng,

Li,

Zhou

et al. 2024

MedComm

View full text Add to dashboard Cite

Gene editing is a growing gene engineering technique that allows accurate editing of a broad spectrum of gene‐regulated diseases to achieve curative treatment and also has the potential to be used as an adjunct to the conventional treatment of diseases. Gene editing technology, mainly based on clustered regularly interspaced palindromic repeats (CRISPR)–CRISPR‐associated protein systems, which is capable of generating genetic modifications in somatic cells, provides a promising new strategy for gene therapy for a wide range of human diseases. Currently, gene editing technology shows great application prospects in a variety of human diseases, not only in therapeutic potential but also in the construction of animal models of human diseases. This paper describes the application of gene editing technology in hematological diseases, solid tumors, immune disorders, ophthalmological diseases, and metabolic diseases; focuses on the therapeutic strategies of gene editing technology in sickle cell disease; provides an overview of the role of gene editing technology in the construction of animal models of human diseases; and discusses the limitations of gene editing technology in the treatment of diseases, which is intended to provide an important reference for the applications of gene editing technology in the human disease.

show abstract